The trial, begun in 2006, involved surgical transplantation of purified neural stem cells into six pediatric patients with the rare and uniformly fatal form of the brain degenerative disorder, Batten disease, plus extensive medical and imaging follow-up.

The cell transplants were also the largest undertaken in clinical trials, ranging from about 300 million to almost a billion cells per patient, and the first time cellular ‘brain transplants’ were utilized to help children.

The definitive publication of the OHSU trial demonstrates that the transplantation operations were all carried out safely at Doernbecher, and that trial patients tolerated the transplanted cells and related medications without serious complications.

OHSU and OHSU Doernbecher researchers and their collaborators at Stanford University and Stem Cells, Inc., the trial sponsor, also discovered that transplanted stem cells appear to survive in the brain for months to years after transplantation, offering hope that in future these types of transplants could help patients with various brain, spinal cord and retinal diseases that are currently untreatable.

Our stem cell transplantation trial was led by OHSU Doernbecher metabolic medicine specialist Robert Steiner, M.D., and myself. I was also privileged to serve as the treating surgeon for each of these six precious patients.

Batten disease is a devastating illness that has already claimed the lives of three of our six study patients, despite their stem cell-based therapy. During the trial, I have had the pleasure of knowing them and their marvelous, compassionate, and truly dedicated families, through their repeated visits to Doernbecher for more than five years.

The entire community of Batten disease families, caretakers, and researchers tremendously values their courage in volunteering to be a part of this groundbreaking attempt to works towards the development of a cure.